University of Kansas Medical Center

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: - To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. - To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. - To characterize the Pompe disease population. - To evaluate the long-term effectiveness of alglucosidase alfa.

Type: Observational [Patient Registry]

Start Date: Sep 2004

open study

Mini percutaneous nephrolithotripsy is the treatment of choice for kidney stones over 2cm. This procedure commonly uses laser energy for breaking the stones, and among possible laser platforms, Hoyag and thulium fiber laser are FDA approved. Recently, this procedure has also included the use of vacuum assisted renal access sheaths, which allows suction to be employed together with the breaking of stones. This study intends to compare outcomes when using either laser platforms with suction sheaths in participants undergoing mini percutaneous nephrolithotripsy for the treatment of kidney stones.

Type: Interventional

Start Date: Aug 2025

open study

TRANSPIRE is an observational study of ~200 individuals who (1) will be initiating gender-affirming hormone therapy (GHT) or (2) are gender minority individuals who do not use GHT. The primary outcome will be to identify how the presence of chronic pain changes overtime with GHT through the use of surveys, quantitative sensory testing (QST), brain MRIs, and qualitative interviews. Following recruitment and consent, participants will complete baseline survey measures and will repeat those measures at 1 months, 3 month, 6 months, and 12 months. QST measures, brain MRIs, and Qualitative Interviews will be offered to participants in cohort (1) and will be completed at baseline and 12 months.

Type: Observational

Start Date: Mar 2025

open study

The goal of this clinical trial is to learn if using a liver-directed therapy with high dose chemotherapy followed by approved cancer treatment to treat patients with colorectal cancer that has spread to the liver is safe and tolerable. The clinical trial will also learn if the liver-directed therapy with high dose chemotherapy works on the disease in the liver. Investigators will compare the use of the liver-directed therapy with high dose chemotherapy followed by approved cancer treatment or approved cancer treatment alone. Participants will: - Undergo up to two liver-directed therapy with high dose chemotherapy procedures followed by approved cancer treatment or take approved cancer treatment alone - Visit clinic at least every two weeks for checkups and tests - Complete scans approximately every two months

Type: Interventional

Start Date: Aug 2025

open study

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

Type: Interventional

Start Date: Jul 2024

open study

The goal of this clinical trial is to test in moderately symptomatic chronic subdural hematoma (CSDH) patients if middle meningeal artery embolization (MMAE) can be used as an alternative to conventional open surgery. The main questions it aims to answer are: - Compared to open conventional surgery, does MMAE reduce the need for rescue surgery or deaths? - What is the safety of MMAE and conventional open surgery in these patients? Participants will be asked to: - Share their medical history and undergo physical examinations - Have blood drawn - Have CT scans of the head - Answer questionnaires - Undergo MMAE or conventional open surgery - Provide information about possible adverse events Researchers will compare participants in the MMAE group with those in the conventional open surgery group to see if there is a reduced need for rescue surgery or deaths and evaluate safety.

Type: Interventional

Start Date: Nov 2024

open study

The purpose of the study is to understand how the study medicine PF-06823859 (dazukibart) works in people with idiopathic inflammatory myopathies (DM and PM). These disorders cause inflammation that weakens the muscles that are important for movement and may also cause skin rash in people with DM. This study is seeking participants who: - Are 18 years of age or older or minimum legal adult age as defined per local regulation, whichever is greater - Have active DM or active PM. - Are receiving a stable dose of 1 corticosteroid taken by mouth and/or 1 traditional immunosuppressant. - Note: Corticosteroids and immunosuppressants are medicines that help reduce inflammation and may signal to the immune system not to attack the body. Dermatomyositis (DM) is a rare disease that causes muscle inflammation that results in muscle weakness and low muscle stamina. Patients with DM have a characteristic skin rash. Polymyositis (PM) is a rare disease that involves mainly muscle inflammation resulting in muscle weakness, that can sometimes be painful. Patients with DM and PM may have trouble going up the steps, walking or getting to a standing position. Some of the participants will receive the study medicine (dazukibart) and some will receive placebo (which is similar to study medicine but contains no medicine in it). The study medicine or placebo will be given as an intravenous (IV) infusion (directly into the veins), which takes about 1 hour; every 4 weeks from Day 1 to Week 48 of the study. Both dazukibart and placebo and will be given at the study site. The study will compare the experiences of people receiving study medication to those of the people who do not. This will help to see if dazukibart is safe and effective. Participants will take part in this study for about 13 months. During this time, participants will have 15 study visits. These visits will be performed at the study site.

Type: Interventional

Start Date: May 2023

open study

The purpose of the Vivistim Registry for Paired VNS Therapy (GRASP) is to gather real-world information on patients with arm and hand deficits post-stroke who are considering Vivistim System treatment. Before and after Vivistim System implant, patient data will be collected and reported throughout the therapy process. Physicians or sites will receive appropriate remuneration for the effort and cost involved in collecting and transmitting this data to MicroTransponder Inc.

Type: Observational [Patient Registry]

Start Date: Mar 2023

open study

The CLAAS® device will be evaluated for safety and efficacy by establishing its performance is non-inferior to the commercially available WATCHMAN® and Amulet™ left atrial appendage closure devices in patients with non-valvular atrial fibrillation. Patients who are eligible for the trial will be randomized to receive either the CLAAS device or the WATCHMAN or Amulet™ devices and will be followed for 5 years after device implant.

Type: Interventional

Start Date: May 2022

open study

This is a multicenter, randomized, open label phase lll trial to assess whether preoperative chemotherapy, as an adjunct to curative-intent surgery, improves the prognosis of high risk DDLPS (dedifferentiated Liposarcoma) and LMS (Leiomyosarcoma) patients as measured by disease free survival. After confirmation of eligibility criteria, patients will be randomized to either the standard arm or experimental arm.

Type: Interventional

Start Date: Jan 2021

open study

Primary Aim: To determine if apixaban is superior to aspirin for prevention of the composite outcome of any stroke (hemorrhagic or ischemic) or death from any cause in patients with recent ICH and atrial fibrillation (AF). Secondary Aim: To determine if apixaban, compared with aspirin, results in better functional outcomes as measured by the modified Rankin Scale.

Type: Interventional

Start Date: Jan 2020

open study

This phase III trial compares the effect of decreased number of radiation (ultra-hypofractionated) treatments to the usual radiation number of treatments (hypofractionation) with standard of care chemotherapy, with cisplatin, gemcitabine or mitomycin and 5-fluorouracil for the treatment of patients with muscle invasive bladder cancer. Hypofractionated radiation therapy delivers higher doses of radiation therapy over a short period of time. Ultra-hypofractionated radiation therapy delivers radiation over an even shorter period of time than hypofractionated radiation therapy. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of tumor cells. Gemcitabine is a chemotherapy drug that blocks the cells from making DNA and may kill tumor cells. Chemotherapy drugs, such as mitomycin-C and 5-fluorouracil (5-FU), work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ultra-hypofractionated radiation may be equally effective as hypofractionated therapy for patients with muscle invasive bladder cancer.

Type: Interventional

Start Date: Oct 2025

open study

This phase Ib/II trial tests the safety, side effects, best dose and how well giving CX-5461 works for the treatment of patients with B-cell non-Hodgkin lymphoma. CX-5461 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving CX-5461 may be safe, tolerable and/or effective in treating patients with B-cell non-Hodgkin lymphoma.

Type: Interventional

Start Date: Oct 2026

open study

The objective of the Safety and Efficacy of Endovascular Treatment of Intracranial Aneurysms with Surpass Elite with GUARDian Flow Diverter (GUARD) trial is to evaluate the safety and efficacy of the Surpass Elite with Guardian Flow Diverter System (FDS) in the treatment of unruptured, wide-neck saccular or fusiform, intracranial aneurysms (IAs) located on the internal carotid artery (ICA) or its branches.

Type: Interventional

Start Date: Jun 2025

open study

Allogeneic transplant is potentially curative for hematological malignancies but its use is limited by the development of GVHD. Ruxolitinib now has FDA approval for treatment of chronic GVHD that has failed 1-2 prior lines of therapy based on a prior large, randomized phase III study. Given this evidence of safety and efficacy in the early refractory setting (after prednisone failure), Ruxolitinib represents an ideal agent to test in the primary therapy setting. Here investigators propose a phase 2 randomized study to compare Ruxolitinib to prednisone as a first-line therapy in the treatment of chronic GVHD.

Type: Interventional

Start Date: Dec 2024

open study

The purpose of the study is to evaluate the clinical safety and efficacy of the Prevail DCB.

Type: Interventional

Start Date: Feb 2025

open study

People who had a stroke at least 6 months prior and who still have difficulty with walking will each be randomly assigned to receive either moderate or vigorous intensity walking exercise. Both protocols will be performed individually with a physical therapist for 45 minutes, 3x/week for 12 weeks. Measures including walking function will be assessed at baseline (PRE), after 4, 8 and 12 weeks of training (12WK) and at 3-month follow up (3moPOST), by raters who are unaware of the participant randomization.

Type: Interventional

Start Date: Feb 2024

open study

Study GLB-001-01 is a first-in-human (FIH), Phase 1, open-label, dose escalation and expansion clinical study of GLB-001 in participants with relapsed or refractory acute myeloid leukemia (R/R AML) or in participants with relapsed or refractory higher-risk myelodysplastic syndromes (R/R HR-MDS). The dose escalation part (Phase 1a) of the study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of GLB-001 administered orally. Approximately 24 participants (up to 42 participants) may be enrolled in Phase 1a of the study. The dose expansion part (Phase 1b) will be followed to understand the relationships among dose, exposure, toxicity, tolerability and clinical activity, to identify minimally active dose, and to select the recommended dose(s) for phase 2 study. Up to 24 participants (12 participants per dose level) may be enrolled in Phase 1b of the study.

Type: Interventional

Start Date: Jan 2024

open study

The purpose of this study is to compare the efficacy and safety of ficlatuzumab plus cetuximab compared to placebo plus cetuximab in participants with recurrent/metastatic (R/M) HPV-negative Head and Neck Cancer. The primary hypothesis is that ficlatuzumab combined with cetuximab is superior to cetuximab alone in terms of progression-free survival and/or overall survival.

Type: Interventional

Start Date: Jan 2024

open study

The safety, tolerability, and antileukemic response of ziftomenib in combination with standard of care treatments for patients with relapsed/refractory acute myeloid leukemia will be examined with the following agents: FLAG-IDA, low-dose cytarabine, and gilteritinib.

Type: Interventional

Start Date: Feb 2024

open study

This Phase III trial will examine the efficacy of computerized cognitive training methods on perceived cognitive impairment in breast cancer survivors.

Type: Interventional

Start Date: Feb 2024

open study

The purpose of this study is to assess the efficacy of metformin to improve airway ion channel function in those with CF-related diabetes (CFRD)

Type: Interventional

Start Date: Feb 2022

open study

The primary aims of this study is to characterize exosomal biomarker levels in patients with locally advanced rectal cancer undergoing neoadjuvant chemoradiation therapy. Compare rates of exosomal expression before during and after chemoradiation therapy with pathologic response rates at the time of APR or LAR. The researchers will also examine the functionality of these exosomes in malignant colonic organoids and mouse models of colorectal cancer.

Type: Observational [Patient Registry]

Start Date: Feb 2018

open study

The intent of this study is to establish a registry of post-surgical outcomes in patients undergoing radical cystectomy at MD Anderson Cancer Center and the collaborating institutions. The goals of this initiative are to obtain a detailed baseline of multiple patient-reported outcomes (PRO) and clinician-reported outcomes (CRO) as well as various presenting conditions associated with them, so that future quality improvement interventions can be evaluated accurately as to their relative contribution to improved outcomes.

Type: Observational [Patient Registry]

Start Date: Apr 2015

open study

The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS. The main questions the trial aims to answer are the following: - Is TRX319 safe when administered to patients with progressive forms of MS? - At what dose does TRX319 work the best to treat participants with primary and or secondary progressive MS? - Is pre-conditioning (with Bendamustine) needed to allow TRX319 to better treat participants with primary and/or secondary progressive MS? Participants will be asked to be on study for up 1 year and may receive up to 3 total administrations of TRX319. While on study, participants will have blood tests and other assessments (MRI scans and lumbar punctures) done to understand the safety of TRX319 and how it may benefit their multiple sclerosis.

Type: Interventional

Start Date: Mar 2026

open study